U.S. Food and Drug Administration Awards Year Two Proceeds from Orphan Products Grant Supporting Palvella Therapeutics’ Phase 3 SELVA Trial of QTORIN™ Rapamycin for Microcystic Lymphatic Malformations

Rhea-AI Summary

Palvella Therapeutics (NASDAQ:PVLA) received Year Two funding from the FDA Office of Orphan Products Development for its Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin for microcystic lymphatic malformations.

The grant provides up to $2.6 million in non-dilutive funding over four years. SELVA is a 24-week, single-arm, baseline-controlled trial that enrolled 51 subjects (target 40) and remains on track for top-line results in Q1 2026. Palvella plans an NDA submission in the second half of 2026 assuming positive results. QTORIN has Breakthrough Therapy, Orphan Drug, and Fast Track designations and could qualify for 7 years of U.S. orphan exclusivity if approved.

Positive

• $2.6M in non-dilutive grant funding over 4 years
• SELVA enrollment exceeded target: 51 vs 40 planned
• Top-line data scheduled for Q1 2026
• Planned NDA submission H2 2026 assuming positive results
• QTORIN has Breakthrough, Orphan, and Fast Track designations

Negative

• None.

Insights

Clinical-Commercial Analyst

FDA awarded year-two orphan grant; Phase 3 fully enrolled and top-line data due in Q1 2026, NDA planned in second half of 2026.

The grant provides up to $2.6 million across the program and validates the clinical design and regulatory interest in QTORIN™ rapamycin. Non-dilutive funding reduces near-term financing pressure while the company completes the 24-week single-arm, baseline-controlled SELVA study that enrolled 51 subjects versus a 40-subject target, and maintains momentum toward top-line data in Q1 2026.

Key dependencies and risks include the actual SELVA top-line outcomes and the FDA's assessment at NDA review; the grant and prior Breakthrough/Orphan/Fast Track designations support regulatory engagement but do not guarantee approval. Watch the announced top-line readout in Q1 2026 and the timing/content of an NDA submission in second half of 2026, which will materially change regulatory clarity and commercial optionality within that 6–12 month horizon.

Second year proceeds granted following FDA review of annual performance progress report on Phase 3 SELVA single-arm, baseline-controlled trial; up to $2.6 million in non-dilutive funding anticipated over the grant period

Top-line data from SELVA expected in the first quarter of 2026; New Drug Application (NDA) submission planned for second half of 2026

WAYNE, Pa., Oct. 13, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced it has received the second year of funding under its FDA Office of Orphan Products Development grant. The grant provides up to $2.6 million in non-dilutive funding over its full 4-year term and supports Palvella’s ongoing Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs).

"We are deeply appreciative of the FDA’s continued support of our Phase 3 program, which reflects the urgency and importance of addressing the significant unmet need faced by the estimated more than 30,000 individuals in the U.S. living with microcystic lymphatic malformations,” said Jeff Martini, Ph.D., Chief Scientific Officer of Palvella. “Exceeding our enrollment goal and maintaining momentum toward top-line data in early 2026 demonstrates the strength of our clinical program and our unwavering commitment to bringing QTORIN™ rapamycin to patients who currently have no FDA-approved therapies.”

The SELVA trial is a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin administered once daily for the treatment of microcystic LMs. Designed to enroll 40 subjects, the trial surpassed its enrollment target, enrolling 51 subjects at leading U.S. vascular anomaly centers. Following an 8-week baseline period and 24-week evaluation, eligible participants may continue treatment in an open-label extension study. The SELVA trial remains on track to report top-line results in the first quarter of 2026. Assuming positive Phase 3 results, Palvella plans to submit an NDA to the FDA in the second half of 2026.

As a part of the Orphan Products Grant Program, Palvella is required to submit an annual performance progress report to the FDA. Following the submission and FDA review of this year’s performance progress report, the FDA granted Palvella the second year of funding. Out of 51 applications received by the FDA Orphan Products Grants Program in fiscal year 2024, Palvella’s Phase 3 SELVA trial was one of only seven new clinical trials selected for funding—and the only Phase 3 trial awarded a grant. The FDA Orphan Products Grants Program provides annual grants to support the development of safe and effective medical products to address unmet medical needs for patients with rare diseases or conditions. Grant applications are individually reviewed and scored for scientific and technical merit by an independent ad hoc panel of rare disease and regulatory experts and may involve consultation with the relevant FDA review division to help determine whether the proposed study will provide acceptable data that could contribute to product approval. Since inception, the program has supported clinical research leading to the approval of more than 85 products.

QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track designations by the FDA for the treatment of microcystic LMs. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S.

About Palvella Therapeutics

Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).

QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.

Forward-Looking Statements

This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.

Contact Information

Investors
Wesley H. Kaupinen
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com

Media
Marcy Nanus
Managing Partner, Trilon Advisors LLC
mnanus@trilonadvisors.com

FAQ

How much funding did Palvella (NASDAQ:PVLA) receive from the FDA Orphan Products grant?

The grant provides up to $2.6 million in non-dilutive funding over a four-year period.

When will Palvella report top-line results from the Phase 3 SELVA trial (PVLA)?

Top-line results from SELVA are expected in Q1 2026.

How many patients enrolled in the Phase 3 SELVA trial for QTORIN (PVLA)?

The SELVA trial enrolled 51 subjects, surpassing its 40-subject enrollment goal.

What regulatory milestones does QTORIN for microcystic LMs (PVLA) currently have?

QTORIN holds Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA.

When does Palvella plan to submit an NDA for QTORIN (PVLA)?

Palvella plans an NDA submission in the second half of 2026, assuming positive Phase 3 results.